FMR1

Lysogene Publishes Positive Preliminary Preclinical Results in the Treatment of Fragile X Syndrome

Retrieved on: 
Thursday, April 21, 2022
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FXS is caused by a mutation of the FMR1 gene that provides instructions for production of the Fragile X Mental Retardation Protein (FMRP).

Key Points: 
  • FXS is caused by a mutation of the FMR1 gene that provides instructions for production of the Fragile X Mental Retardation Protein (FMRP).
  • Ralph Laufer, CSO of Lysogene, commented: These preclinical results confirm the validity of our innovative approach targeting DGKk for the treatment of FXS, a CNS pathology with high unmet medical need.
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).
  • Lysogene is also developing an innovative AAV gene therapy approach for the treatment of Fragile X syndrome, a genetic disease related to autism.

Zynerba Pharmaceuticals Announces European Commission Has Granted Orphan Drug Designation for Zygel™ in Fragile X Syndrome

Retrieved on: 
Monday, February 28, 2022
GLOBE, Annual report, Intellectual, Fragile X syndrome, European Medicines Agency, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Twitter, ASD, Growth, Autism, Fast Track, FXS, Prevalence, European Commission, Diagnosis, Security (finance), COVID-19, Company, Cannabidiol, United Kingdom, Royal commission, Infection, Disease, DiGeorge syndrome, U.S. Securities and Exchange Commission, FMR1, EC, Food, Intellectual disability, Methylation, Private Securities Litigation Reform Act, Skin, FDA, Nasdaq, EMA, Patient, Marketing, Union, Form 10-K, Pharmaceutical industry, Medicine, EU, Jean-François Zygel

Orphan drug designation by the EC is another important milestone for Zygel, said Armando Anido, Chairman and Chief Executive Officer of Zynerba.

Key Points: 
  • Orphan drug designation by the EC is another important milestone for Zygel, said Armando Anido, Chairman and Chief Executive Officer of Zynerba.
  • As previously disclosed, Zygel has also been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of FXS and 22q11.2 deletion syndrome.
  • Additionally, the FDA has granted Fast Track designation for Zygel for the treatment of behavioral symptoms associated with FXS.
  • Zygel is an investigational drug product in development for the potential treatment of behavioral symptoms associated with Fragile X syndrome (FXS), autism spectrum disorder (ASD), and 22q11.2 deletion syndrome (22q).

Nova Mentis Study Confirms Oral Microdose Psilocybin Treatment of Autism

Retrieved on: 
Wednesday, February 16, 2022
Psilocybin, CTA, Fragile X syndrome, Roma Tre University, Family, Observational study, CEO, Nova, Finder, Company, Science, Human, Autism, Brain, Animal, FXS, Canadian Securities Exchange, Neurodevelopmental disorder, Health Canada, Therapy, RATS, FSE, Government budget balance, View, FMR1, NOVA, CSE, Placement, Multimedia, ASD, News, Manduca sexta, Autism spectrum, Industry, Coronavirus Scientific Advisory Board (Turkey), Pharmaceutical industry, BC

VANCOUVER, BC, Feb. 16, 2022 /PRNewswire/ -Nova Mentis Life Science Corp. (CSE: NOVA) (FSE: HN3Q) (OTCQB: NMLSF) ("NOVA" or the "Company"), a biotechnology company and global leader in first-in-class psilocybin-based therapeutics and complementary diagnostics for neuroinflammatory disordersis pleased to announce that Nova Mentis successfully completed an oral microdose psilocybin preclinical study, in the laboratory of Dr. Viviana Trezza, Rome Tre University, Rome, Italy.

Key Points: 
  • VANCOUVER, BC, Feb. 16, 2022 /PRNewswire/ -Nova Mentis Life Science Corp. (CSE: NOVA) (FSE: HN3Q) (OTCQB: NMLSF) ("NOVA" or the "Company"), a biotechnology company and global leader in first-in-class psilocybin-based therapeutics and complementary diagnostics for neuroinflammatory disordersis pleased to announce that Nova Mentis successfully completed an oral microdose psilocybin preclinical study, in the laboratory of Dr. Viviana Trezza, Rome Tre University, Rome, Italy.
  • The recent oral microdose data set not only confirms but exceeds our original injectable formulation results," says Will Rascan, CEO of NOVA.
  • "The clear positive data is critical as we prepare to submit ourclinical trial application to Health Canada for a Phase 2A study evaluating psilocybin microdose therapy for fragile X syndrome."
  • The aim of the present study was to evaluate various oral doses of the company's proprietary psilocybin in a rat model of FXS.

Fragile X Syndrome Market Spotlight Report 2021: 10-year Disease Incidence and Prevalence Forecasts, Probability of Success, Licensing and Asset Acquisition Deals, Drug-specific Revenue Forecasts - ResearchAndMarkets.com

Retrieved on: 
Thursday, January 27, 2022
Science, Other Science, Research, Managed Care, General Health, Health, Mental Health, Other Health, FXS, FXTAS, Fragile X syndrome, Turner syndrome, Government budget balance, Novartis, FMRP, Prevalence, FMR1, Therapy, Genetic disorder, Clinical trial, Potassium, Probability, Learning, Mutation, Residential care, Pharmaceutical industry

The "Market Spotlight: Fragile X Syndrome" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Market Spotlight: Fragile X Syndrome" report has been added to ResearchAndMarkets.com's offering.
  • Fragile X syndrome (FXS) is a genetic disorder leading to various developmental problems ranging from learning disabilities to cognitive impairment.
  • FXS is caused by mutation of the fragile X mental retardation 1 (FMR1) gene, which usually makes a protein known as fragile X mental retardation protein (FMRP).
  • Individuals who have other fragile X-associated disorders (FXTAS) have mutations in their FMR1 gene, but they generally make some FMRP.

Bionano Genomics Announces Publication of Interim Readout from the Consortium Conducting a Postnatal Clinical Trial Intended to Support Establishing Optical Genome Mapping as Part of Standard of Care in Genetic Disease Diagnosis

Retrieved on: 
Monday, January 10, 2022
Clinical trial, OGM, SAN, DNA, Standard of care, SOC, Biology, Security (finance), Genetic testing, Columbia University Irving Medical Center, Karyotype, Lymphatic system, Cancer, Patient, TAT, Genome, FACMG, Lineagen, CMA, In situ hybridization, U.S. Securities and Exchange Commission, FMR1, Forward-looking statement, Annual report, Lists of diseases, Program, Genomics, University of Rochester Medical Center, Solution, Workflow, Data analysis, Research, Medical College of Wisconsin, Company, Diagnosis, Private Securities Litigation Reform Act, Doctor of Philosophy, Software, University, Autism, FISH, Medicine, COVID-19, Hospital Insurance and Diagnostic Services Act, Medical college, GLOBE, Fluorescence, IRB, Axis and Soviet air operations during Operation Barbarossa, Institutional review board, Adoption, Medical imaging, Pharmaceutical industry, Medical device

SAN DIEGO, Jan. 10, 2022 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (BNGO), pioneer of optical genome mapping (OGM) solutions on the Saphyr® system and provider of NxClinical™, the leading software solutions for visualization, interpretation and reporting of genomic data, today announced the publication of the first readout from the ongoing clinical trial designed to support establishing OGM as part of standard of care (SOC) in diagnosis of genetic disease for postnatal patients. This publication reports on the postnatal genetic disease diagnostic arm of Bionano’s study to evaluate OGM as an alternative to SOC workflows in four key clinical areas: prenatal and postnatal genetic diseases, hematologic malignancies and solid tumors. The studies will compare OGM to SOC, including concordance, reproducibility, technical success rates, turnaround time (TAT), diagnostic yield, health economics and patient outcomes. This first interim readout is designed to evaluate endpoints connected to analytical performance in key areas of technical performance and reproducibility of OGM.

Key Points: 
  • This first interim readout is designed to evaluate endpoints connected to analytical performance in key areas of technical performance and reproducibility of OGM.
  • The study is an Institutional Review Board-approved, multicenter, double-blinded trial with 202 clinical research subjects analyzed in a total of 331 sample runs.
  • All samples had been previously tested with traditional methods like karyotyping, fluorescence in situ hybridization (FISH) and chromosomal microarray (CMA).
  • The samples were from cases with a genetic diagnosis (152), cases without a genetic diagnosis (6) and controls (44).

Zynerba Pharmaceuticals Announces Clinical Development Updates for FXS, ASD, 22q and DEE

Retrieved on: 
Tuesday, January 4, 2022
CONNECT, The Sarah Jane Brain Foundation, EMA, Food, Program, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, European Medicines Agency, María Luisa Anido, Â, Methylation, Company, Behavior, Patient, INSPIRE, DiGeorge syndrome, Irritability, ASD, Investigational New Drug, Laboratory, Fragile X syndrome, Automated ECG interpretation, FXS, Marketing, FDA, GLOBE, Autism, Union, FMR1, DEE, Nasdaq, Vital signs, MAA, Pharmaceutical industry, Medical device, Jean-François Zygel, EU

DEVON, Pa., Jan. 04, 2022 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today provided program updates for Fragile X syndrome (FXS), autism spectrum disorder (ASD), 22q11.2 deletion syndrome (22q) and developmental and epileptic encephalopathies (DEE). In addition to the continued clinical development of Zygel in FXS, the Company plans to focus on the development of Zygel in ASD and 22q. Based on Company research and strategic prioritization, the decision has been made to not move forward in DEE at this time.

Key Points: 
  • Company plans to focus development of Zygel in FXS, ASD and 22q
    Zynerba to hold conference call tomorrow, January 5, 2022 at 9:00 a.m.
  • In addition to the continued clinical development of Zygel in FXS, the Company plans to focus on the development of Zygel in ASD and 22q.
  • Based on Company research and strategic prioritization, the decision has been made to not move forward in DEE at this time.
  • Zynerbamanagement will host a live conference call and webcast tomorrow, January 5, 2022, at 9:00 a.m. Eastern Timeto discuss updates to its clinical development plans.

Zynerba Pharmaceuticals Reports Second Quarter 2021 Financial Results and Operational Highlights

Retrieved on: 
Monday, August 9, 2021
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The trial is designed to confirm the positive results observed in a population of responders in the Companys previously conducted CONNECT-FX trial.

Key Points: 
  • The trial is designed to confirm the positive results observed in a population of responders in the Companys previously conducted CONNECT-FX trial.
  • ( Press release )
    Research and development expenses were $5.5 million for the second quarter of 2021, including stock-based compensation of $1.0 million.
  • General and administrative expenses were $4.4 million in the second quarter of 2021, including stock-based compensation expense of $0.9 million.
  • The net loss for the second quarter of 2021 was $10.0 million, with basic and diluted loss per share of $(0.25).

Lysogene Enters into an Exclusive Worldwide License Agreement with SATT Conectus for a Gene Therapy Candidate for the Treatment of the Fragile X Syndrome

Retrieved on: 
Friday, June 11, 2021
Biotechnology, Manufacturing, Other Health, Health, Pharmaceutical, Other Science, Research, Other Manufacturing, Genetics, Science, Clinical trials, Branches of biology, Biology, Clinical medicine, Intellectual disability, Biotechnology, FMR1, Fragile X syndrome, Gene therapy, Fragile X-associated tremor/ataxia syndrome, FXR2

Lysogene (FR0013233475 LYS) (Paris:LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, today announces that it has entered into an exclusive, worldwide license agreement with SATT Conectus for the development and commercialization of a gene therapy candidate for the treatment of Fragile X syndrome.

Key Points: 
  • Lysogene (FR0013233475 LYS) (Paris:LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, today announces that it has entered into an exclusive, worldwide license agreement with SATT Conectus for the development and commercialization of a gene therapy candidate for the treatment of Fragile X syndrome.
  • The gene therapy drug candidate aims at compensating for reduced DGKk, a novel therapeutic target whose synthesis is regulated by Fragile X Mental Retardation Protein (FMRP), the missing protein responsible for Fragile X syndrome.
  • Karen Aiach, Founder, Chairman and CEO of Lysogene commented: The acquisition of this global license for Fragile X syndrome is a major step forward.
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).